IACC Strategic Plan Update Question 4 Planning Group Conference Call - November 13, 2013
Topic | Topic Description |
---|---|
Date: | Wednesday, November 13, 2013 |
Time: | 2:30 p.m. to 4:30 p.m. Eastern |
Agenda: | The planning group for Question 4 (Treatments and Interventions) will discuss updates for the IACC Strategic Plan. |
Place: | No in-person meeting; conference call only |
Conference Call: | Dial: (800) 857-9611 Access code: 6269192 |
Materials: | Meeting materials |
Contact Person: | Ms. Lina Perez Office of Autism Research Coordination National Institute of Mental Health, NIH 6001 Executive Boulevard, NSC, Room 6182A Rockville, Maryland 20852 Phone: (301) 443-6040 E-mail: IACCPublicInquiries@mail.nih.gov |
Please Note: | The conference call will be open to the public in listen-only mode. Members of the public who participate using the conference call phone number will be able to listen to the meeting but will not be heard. If you experience any technical problems with the conference call, please e-mail HelpDeskIACC@gmail.com or call the IACC Technical Support Help Line at 415-652-8023. Accommodations Statement: The meeting will be open to the public via conference call. Individuals who participate by using this electronic service and who need special assistance such as captioning or other reasonable accommodations should submit a request to the Contact Person listed on this notice at least 3 days prior to the meeting. Schedule subject to change. |
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No in-person meeting; conference call only. The materials for the meeting can be found here.
Time | Event |
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2:30 p.m. | Roll Call and Opening Remarks Thomas Insel, M.D. Director, National Institute of Mental Health (NIMH) Chair, IACC Susan Daniels, Ph.D. Acting Director, Office of Autism Research Coordination, NIMH Executive Secretary, IACC |
2:40 p.m. | Discussion of Progress Toward Meeting Strategic Plan Question 4 Objectives – "Which Treatments and Interventions Will Help?" (Treatments and Interventions) |
3:55 p.m. | Discussion of Progress Toward Meeting Question 4 Aspirational Goal: "Interventions Will Be Developed That Are Effective For Reducing Both Core And Associated Symptoms, For Building Adaptive Skills, And For Maximizing Quality Of Life And Health For People With ASD." |
4:25 p.m. | Wrap-up and Next Steps |
4:30 p.m. | Adjournment |
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- Roll Call and Opening Remarks
- Discussion of Question 4 IACC Portfolio Analysis Documents and Strategic Plan Progress
The Interagency Autism Coordinating Committee (IACC) Subcommittee for Basic and Translational Research Strategic Plan for Autism Spectrum Disorder, Question 4 Planning Group, convened a conference call on Wednesday, November 13, 2013, from 2:36 p.m. to 4:30 p.m.
In accordance with Public Law 92-463, the meeting was open to the public. Susan Daniels, Ph.D., Executive Secretary, IACC, presided.
Participants:
Susan Daniels, Ph.D., Executive Secretary, IACC, Office of Autism Research Coordination (OARC), (NIMH); Idil Abdull, Somali American Autism Foundation; Tiffany Farchione, M.D., U.S. Food and Drug Administration (FDA); Lisa Gilotty, Ph.D., NIMH (representing Thomas Insel, M.D.); Jeremy Veenstra-VanderWeele, M.D., Vanderbilt University; Paul Wang, M.D., Autism Speaks
Roll Call and Opening Remarks
Dr. Susan Daniels welcomed the Planning Group and members of the public, and called roll. The external experts briefly introduced themselves. Dr. Daniels said that this Group was charged with the update for Question 4 (Which Treatments and Interventions Will Help?) of the IACC Strategic Plan. She noted that the goal of this call of the Planning Group was to qualitatively evaluate the scientific progress made on the Question 4 objectives, and to identify any gaps with the help of external experts. In addition, the Group was to discuss progress made toward the aspirational goals of Questions 4.
Discussion of Question 4 IACC Portfolio Analysis Documents and Strategic Plan Progress
Dr. Daniels reviewed meeting materials prepared by the Office of Autism Research Coordination to provide funding and project information for Planning Group reference. All of the materials are available online. The materials included: a Compiled Objectives (cumulative funding) Table, a Project Data Table (2008-2010), and a Conclusions Table. The Compiled Objectives (cumulative funding) Table showed the alignment of funding across years (2008-2012) for each objective in Question 4. The Project Data Table (made available for the previous conference call) was provided to the Committee members, and is available online. These Tables included some live links for 2008-2010 projects. The Conclusions Table was a compilation of initial determinations about the funding progress on Question 4 objectives made by Question 4 Planning Group members during the previous conference call. These tables provided brief summaries of the discussion of each objective.
Dr. Daniels guided the Group through discussions of research progress, gaps, and barriers for each of the objectives. She began each discussion by reviewing the overall conclusions from the previous Planning Group call regarding funding, projects, and conclusions.
4.S.A Support at least three randomized controlled trials that address co-occurring medical conditions associated with ASD by 2010. On the previous call, the Group had agreed that the recommended budget had been met. However, they considered the funded three projects (sleep, anxiety, and seizures) as a start to the needed research. Dr. Jeremy Veenstra-VanderWeele said that most of the work appeared to be the adaption of interventions used in the absence of autism. He said that there had been quite a lot of research on ADHD, but more needed to be known about other areas, like sleep problems. He added that core symptoms were very challenging, and that perhaps funding for research on non-core symptoms would be better spent on core symptoms. Dr. Paul Wang agreed, but said that a large number of medications were being prescribed as treatments for non-core symptoms. It was important to understand the efficacy and safety of these treatments. He said that sleep was particularly important in his opinion, but it was unclear whether these projects spanned the age spectrum. Dr. Veenstra-VanderWeele said that pediatric sleep treatments did not necessarily translate to children with autism. The same was true for anxiety. These areas needed work. Ms. Idil Abdull asked what she could do to help her son with sleep problems – waking several times during the night. She said that her perception was that money had been spent on research, but it had not resulted in any interventions or treatments for families. Dr. Veenstra-VanderWeele said that he was asked this question frequently, and he would like to have answers to give to families. More is known about sleep initiation, and less about nighttime awakenings from sleep.
4.S.B Standardize and validate at least 20 model systems (e.g., cellular and/or animal) that replicate features of ASD and will allow identification of specific molecular targets or neural circuits amenable to existing or new interventions by 2012. The Group previously had agreed that recommended budget had been met and exceeded. Dr. Daniels said that some of the members on the previous call were concerned this area had been too highly prioritized. Dr. Veenstra-VanderWeele said that only now were there appropriate animal and cellular models for autism due to the investment that had been made in this area. Given the state of the knowledge, it made sense that there was a lot of funding in this area, and that this level of funding should continue. These models often did not yield results for many years, so a steady investment was required. He pointed out that it had taken 20 years for research to move from in vitro and animal models to clinical trials of potential treatments for symptoms of Fragile X. Dr. Wang said that it was important not to expect too much from animal models, as the results from animal models are not always translated to humans. While there are animal models that mimic certain symptoms of autism, many aspects of autism cannot be captured perfectly in an animal model. Dr. Tiffany Farchione said that animal models were useful as preclinical toxicology models.
4.S.C Test safety and efficacy of at least five widely used interventions (e.g., nutrition, medications, assisted technologies, sensory integration, medical procedures) that have not been rigorously studied for use in ASD by 2012. The Group had agreed previously that the recommended budget had been partially met, but more work was needed. They had noted that this was an area of great public interest. Ms. Abdull asked for the experts to discuss nutritional interventions and sensory processing/integration. Dr. Veenstra-VanderWeele said that there was a carefully conducted study of a gluten-free and casein-free (GFCF) diet by Susan Hyman and colleagues at University of Rochester that had not found any statistically-significant benefits to the GFCF diet for children with autism.1, 2 Dr. Wang said in light of recent research showing that individuals with ASD had higher rates of antibodies against wheat components,3 more research was warranted. With regard to sensory integration, Dr. Veenstra-VanderWeele said that a study was needed that carefully tested a manualized intervention, and provided clear evidence whether the intervention was helpful. He added that in general, there was so much heterogeneity in this area that it was hard to identify next steps. It was important that future studies be well designed and adequately powered to detect a difference, and provide clear answers about interventions. Dr. Wang said that the challenge was designing good scientific studies. There was a brief discussion of funding recommendations in the Strategic Plan and the NIH funding process.
4.S.D Complete two multi-site randomized controlled trials of comprehensive early intervention that address core symptoms, family functioning and community involvement by 2013. On the last call, the Group agreed that the recommended budget had been met and exceeded. They noted that most studies tended to be small and underpowered. Dr. Veenstra-VanderWeele said that studies were needed, which were adequately powered to answer questions. Ms. Abdull noted that more than half of the states used for Applied Behavior Analysis (ABA) intervention because there was research to support this intervention. However, it was not appropriate for older children. Research was needed to provide evidence for a broader range of interventions that are effective across different parts of the lifespan. Evidence was also needed for community/family involvement. Dr. Veenstra-VanderWeele said that this objective included several different goals and should be broken down.
4.S.E Convene a workshop to advance the understanding of clinical subtypes and treatment personalization (i.e., what are the core symptoms to target for treatment studies) by 2011.
Dr. Wang said that there had been a workshop on outcome measures that was somewhat related. Dr. Veenstra-VanderWeele noted that the Foundation for NIH had been working on a biomarkers initiative, which was oriented toward treatments, and also might be related.
4.S.F Launch randomized controlled trials of interventions including biological signatures and other measures to predict response, and monitor quality of life and functional outcomes in each of the following groups: five trials in infants and toddlers by 2013; three trials in school-aged children and/or adolescents by 2013; three trials in adults by 2014. Previously the Group agreed that the recommended budget had been partially met. More work was needed. Again, trials were small, and perhaps were underpowered. Dr. Daniels and Dr. Lisa Gilotty briefly discussed coding issues, and the potential for overlap of questions and projects. Dr. Veenstra-VanderWeele said that the field was moving closer to using behavioral signatures and other indicators to personalize treatment. Small pilot studies were necessary before moving on to large trials. This area was expected to develop more in the next 5 years. Dr. Wang said that future randomized controlled trials (RCTs) would include biological assays.
4.S.G Support at least five studies on interventions for nonverbal individuals with ASD by 2012. Such studies may include: projects examining service-provision models that enhance access to augmentative and alternative communication (AAC) supports in both class room and adult service-provision settings, such as residential service-provision and the impact of such access on quality of life, communication, and behavior; studies of novel treatment approaches that facilitate communication skills in individuals who are nonverbal, including the components of effective AAC approaches for specific subpopulations of people with ASD; and studies assessing access and use of AAC for children and adults with ASD who have limited or partially limited speech and the impact on functional outcomes and quality of life. The Group felt that the recommended budget had been met, but agreed that work needed to continue in this area. Dr. Veenstra-VanderWeele said that verbal ability was an important area of research, particularly for families. Encouragingly, work was growing. Dr. Wang added that more focus was needed on individuals with more severe levels of disability.
4.S.H Support at least two studies that focus on research on health promotion and prevention of secondary conditions in people with ASD by 2012. Secondary conditions of interest include weight issues and obesity, injury, and co-occurring psychiatric and medical conditions. On the last call, the Group agreed that the recommended budget had been partially met, but additional work was needed to address some specific issues in this objective. Dr. Daniels noted that this objective overlapped with Question 5. Dr. Veenstra-VanderWeele said that this objective also overlapped with 4.S.A. He was unsure how psychiatric and medical conditions could be secondary. Dr. Wang mentioned an ongoing study4 on the use of metformin to treat young people with ASD, who were overweight as a result of treatment with antipsychotic medication. However, he did not see this study listed under this objective. Dr. Veenstra-VanderWeele noted that the study was included under objective 4.S.A., but could fit both objectives 4.S.A. and 4.S.H. He also said that co-occurring medical and psychiatric conditions really did not fit into health promotion and prevention of secondary conditions. Dr. Daniels suggested that the group's recommendation could be to remove the portion about secondary conditions and to keep the objective focused on health promotion and prevention. Dr. Veenstra-VanderWeele agreed, saying that it would be most appropriate to focus the objective more clearly on health promotion and prevention, which is a gap that is not covered in other parts of the Strategic Plan.
4.L.A Complete at least three randomized controlled trials on medications targeting core symptoms in people with ASD of all ages by 2014. The Group agreed on the last call that the recommended budget had been partially met, but studies tended to be small. More investment was needed in order to produce meaningful results. It was noted that the funding for this objective did not include investment from pharmaceutical companies or funding from smaller family foundations.
Dr. Wang said that the field had been laying the foundation for this objective, and would become very engaged in the future. Animal efficacy and safety studies were needed before moving into human trials. This was an area were momentum was building, as this type of data accumulated. In addition, studies in autism were already underway for drugs previously approved for other indications. These drugs didn't require preclinical work. Dr. Veenstra-VanderWeele added that previous and current studies would be small. Dr. Tiffany Farchione agreed. FDA was seeing small proof-of-concept studies, particularly ones addressing core symptoms. Dr. Veenstra-VanderWeele said that once there were treatments that showed efficacy, follow-on work would help identify measures of change for core symptoms.
4.L.B Develop interventions for siblings of people with ASD with the goal of reducing the risk of recurrence by at least 30% by 2014. The Group agreed during the last call that the recommended budget had been only partially met, and only a small number of projects had been funded. They felt that the intent of the objective had not been met. Dr. Veenstra-VanderWeele said that this was an encouraging area. There were a number of studies of early intensive behavioral interventions, as well as studies showing that sibling risk was greater than previously thought. This area of research was ready for a significant increase in funding. Ms. Abdull said that this was a very important issue in the community. Some families had more than one child with ASD. Early intervention did not mitigate all of the symptoms of autism in siblings. Prevention research was essential, she said.
4.L.C Conduct at least one study to evaluate the safety and effectiveness of medications commonly used in the treatment of co-occurring conditions or specific behavioral issues in people with ASD by 2015. The Group agreed that the recommended budget had been partially met. Dr. Veenstra-VanderWeele said funding for this area was too low. He noted that there were many off-label studies of drugs approved for other indications – such as ADHD. However, other co-occurring conditions – sleep, anxiety, etc. – were greater priorities. Dr. Wang added that it was important to study the efficacy of drugs that were being used in the clinic off-label in the ASD population. Ms. Abdull agreed that many potentially unsafe products were being marketed as treatments. Large studies were needed to identify safe and efficacious treatments. This information would be very useful to parents.
4.L.D Support at least five community-based studies that assess the effectiveness of interventions and services in broader community settings by 2015. Such studies may include comparative effectiveness research studies that assess the relative effectiveness of: different and/or combined medical, pharmacological, nutritional, behavioral, service-provision, and parent- or caregiver-implemented treatments; scalable early intervention programs for implementation in underserved, low-resource, and low-literacy populations; and studies of widely used community intervention models for which extensive published data are not available. Outcome measures should include assessment of potential harm as a result of autism treatments, as well as positive outcomes. The Group previously agreed that the recommended budget had been partially met. More projects had been supported than specified in the objective. Despite this, they felt that projects did not cover the scope of interventions in the community. Dr. Veenstra-VanderWeele said that there emphasis was on interventions that are being implemented but without evidence. It was important to translate interventions that were efficacious an academic setting to the community. Not enough of this work was being done. Ms. Abdull asked for recommendations regarding disparities in underserved communities. Dr. Veenstra-VanderWeele said that evidence-based treatments should be evaluated to determine if they could be used in a variety of settings. Dr. Gilotty noted that a set of RFAs had been released that focused on underserved communities. The Group briefly discussed coordination of services.
Aspirational goal: Interventions will be developed that are effective for reducing both core and associated symptoms, for building adaptive skills, and for maximizing quality of life and health for people with ASD. Ms. Abdull said that research was needed on tools and techniques to help children who were minimally-verbal to communicate. In particular, evidence for existing interventions would be very useful with regard to obtaining insurance and Medicaid coverage. Dr. Veenstra-VanderWeele said that the Early Start Denver Model5 was one of the key advances in the treatment realm in the last 5 years. In addition, cognitive behavioral therapies – primarily implemented for more verbal children with typically higher IQs – were another area of progress. Significant evidence backed the efficacy of those therapies for anxiety. He added that there was some evidence for social skills treatments as well. In terms of medical treatments, there was evidence for the efficacy of aripiprazole.6 Evidence was also accumulating for medications to treat ADHD. However, the overall objectives were similar to those from 5 years ago. Dr. Wang noted that early manifestations changed with time. Early deficits or impairments could manifest later in diverse ways. It might be that the same was true of treatments. Those that addressed early core symptoms could have cascading benefits. Potentially, if the core symptoms improved, some of the associated symptoms would improve―adaptive skills would improve, quality of life would improve, and secondary health issues would improve. Dr. Veenstra-VanderWeele said that the field had shifted developmentally. There were opportunities where there hadn't been before. In particular, new animal and cellular models presented opportunities for translation. Dr. Daniels provided an overview of the next steps and the upcoming workshop.
Adjournment
The conference call was adjourned at 4:30 p.m.
Certification
I hereby certify that this meeting summary is accurate and complete.
/Susan Daniels/ November 22, 2013
Susan A. Daniels, Ph.D.
Executive Secretary, Interagency Autism Coordinating Committee
References
1 University of Rochester Medical Center. Research: Dietary Treatment of Young Children with Autism: Behavioral Effects of the Gluten Free and Casein Free Diet.
2 University of Rochester Medical Center Press Release. Popular Autism Diet Does Not Demonstrate Behavioral Improvement
3 Lau NM, Green PH, Taylor AK, et al. Markers of Celiac Disease and Gluten Sensitivity in Children with Autism. PLoS One. 2013 Jun 18;8(6):e66155. [PMID: 23823064]
4 Anagnostou E. Treatment of Overweight Induced by Antipsychotic Medication in Young People With Autism Spectrum Disorders (ASD) (MET). In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000 - [2014 May 19]. Available from: NLM Identifier: NCT 01825798.
5 Dawson G, Rogers S, Munson J, et al. Randomized, controlled trial of an intervention for toddlers with autism: the Early Start Denver Model. Pediatrics. 2010 Jan;125(1):e17-23. [PMID: 19948568]
6 Ching H, Pringsheim T. Aripiprazole for autism spectrum disorders (ASD). Cochrane Database Syst Rev. 2012 May 16;5:CD009043. [PMID: 22592735]
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- 2012 IACC Strategic Plan Update
- 2011 IACC Strategic Plan
- Compiled Objective Chart for Question 4 2008 to 2012 (PDF – 170 KB)
- Conclusions by Objective for Question 4 (PDF – 143 KB)
Portfolio Analysis
- 2012 Portfolio Analysis Question 4 Project Listing (PDF – 653 KB)
- 2011 Portfolio Analysis Question 4 Project Listing (PDF – 635 KB)
Conclusions by Objective for Question 4 (Treatments and Interventions)
Prepared for IACC Strategic Plan Update Question 4 Planning Group Conference Call November 13, 2013
IACC Strategic Plan Objectives | Planning Group Summary | Funding 2008-2013 |
---|---|---|
IACC Strategic Plan Objectives4.S.A Support at least three randomized controlled trials that address co-occurring medical conditions associated with ASD by 2010. |
Planning Group SummaryThe recommended budget was met, and more than 3 projects were funded, but these projects are just a start on what needs to be done. Projects include trials of sleep interventions, cognitive behavioral therapy for anxiety, and treatments for seizure. However, more work is needed to address those co-occurring conditions more thoroughly and to address other co-occurring conditions. |
Funding 2008-2013$17,105,378 |
IACC Strategic Plan Objectives4.S.B Standardize and validate at least 20 model systems (e.g., cellular and/or animal) that replicate features of ASD and will allow identification of specific molecular targets or neural circuits amenable to existing or new interventions by 2012. |
Planning Group SummaryThe recommended budget was met and exceeded, and more than 90 projects were supported to develop animal models. Planning Group members questioned whether this area is being too highly prioritized in comparison to other areas that are more readily applicable to current needs . |
Funding 2008-2013$102,110,669 |
IACC Strategic Plan Objectives4.S.C Test safety and efficacy of at least five widely used interventions (e.g., nutrition, medications, assisted technologies, sensory integration, medical procedures) that have not been rigorously studied for use in ASD by 2012. |
Planning Group SummaryThe recommended budget was partially met and several projects were funded in this area, but more work and funding are needed, as this is an area of significant public interest. The group noted that interventions for minimally verbal children are needed; some projects on assistive communication technologies and robotics and speech processing technology to assist with social communication training are funded, but more are needed. There are other projects related to minimally verbal autism in objective 4SG. |
Funding 2008-2013$8,946,921 |
IACC Strategic Plan Objectives4.S.D Complete two multi-site randomized controlled trials of comprehensive early intervention that address core symptoms, family functioning and community involvement by 2013. |
Planning Group SummaryThe recommended budget was met and exceeded. In 2011 and 2012, ~20 trials were supported, but it appears that these trials are small and underpowered. Larger trials are more likely to produce definitive results. |
Funding 2008-2013$42,088,407 |
IACC Strategic Plan Objectives4.S.E Convene a workshop to advance the understanding of clinical subtypes and treatment personalization (i.e., what are the core symptoms to target for treatment studies) by 2011. |
Planning Group SummaryA workshop on this workshop has not taken place, based on information that is currently available. |
Funding 2008-2013$0 |
IACC Strategic Plan Objectives4.S.F Launch randomized controlled trials of interventions including biological signatures and other measures to predict response, and monitor quality of life and functional outcomes in each of the following groups:
|
Planning Group SummaryThe recommended budget has been partially met. The investment in projects under this objective is making good progress toward the recommended amount, with more than 20 projects funded in 2011 and 2012; however, more work is needed. It appears the trials may be too small and underpowered. |
Funding 2008-2013$41,177,035 |
IACC Strategic Plan Objectives4.S.G Support at least five studies on interventions for nonverbal individuals with ASD by 2012. Such studies may include:
|
Planning Group SummaryThe recommended budget has been met and 11-16 studies were funded in 2010-2012, but work needs to continue in this area. |
Funding 2008-2013$9,580,403 |
IACC Strategic Plan Objectives4.S.H Support at least two studies that focus on research on health promotion and prevention of secondary conditions in people with ASD by 2012. Secondary conditions of interest include weight issues and obesity, injury, and co-occurring psychiatric and medical conditions. |
Planning Group SummaryThe recommended budget was partially met and a small number of projects were funded, but further work is needed to address some of the specific issues described in the objective. |
Funding 2008-2013$1,404,969 |
IACC Strategic Plan Objectives4.L.A Complete at least three randomized controlled trials on medications targeting core symptoms in people with ASD of all ages by 2014. |
Planning Group SummaryThe recommended budget has only partially been met. Ten-fourteen studies have been funded, but they are small and likely underpowered studies. More investment is needed on these kinds of projects in order to get meaningful results. |
Funding 2008-2013$9,715,095 |
IACC Strategic Plan Objectives4.L.B Develop interventions for siblings of people with ASD with the goal of reducing the risk of recurrence by at least 30% by 2014. |
Planning Group SummaryThe recommended budget has only partially been met and only a small number of projects funded. The intent of the objective has not been met. |
Funding 2008-2013$831,111 |
IACC Strategic Plan Objectives4.L.C Conduct at least one study to evaluate the safety and effectiveness of medications commonly used in the treatment of co-occurring conditions or specific behavioral issues in people with ASD by 2015. |
Planning Group SummaryThe recommended budget was partially met. A small number (3-7) of studies of pharmacological interventions was funded. |
Funding 2008-2013$6,475,421 |
IACC Strategic Plan Objectives4.L.D Support at least five community-based studies that assess the effectiveness of interventions and services in broader community settings by 2015. Such studies may include comparative effectiveness research studies that assess the relative effectiveness of:
IACC Recommended Budget: $37,500,000 over 5 years |
Planning Group SummaryThe recommended budget has been partially met and 30-45 studies have been supported, exceeding the initial target. Considerable work has been done under this objective, but these projects do not cover the scope of interventions in the community. |
Funding 2008-2013$25,239,169 |
IACC Strategic Plan ObjectivesNot specific to any objective |
Planning Group Summary
|
Funding 2008-2013$44,566,554 |
IACC Strategic Plan ObjectivesTotal funding for Question 4 |
Planning Group Summary
|
Funding 2008-2013$309,241,132 |
This document is for discussion purposes only and does not reflect the decisions of the IACC
Highlighting of each total gives an indication of the progress toward meeting the IACC recommended budget for each objective. Green highlighting indicates that funding fully meets the recommend budget. Yellow highlighting denotes that funding for a particular objective partially meets the IACC recommended budget, while red highlighting indicates that there has been no funding towards the particular objective.
blue text is an insertionred text is a deletion
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- Meeting Transcript (PDF – 200 KB)
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